Evaluating HSK39297 for treating Paroxysmal Nocturnal Hemoglobinuria
A Multicenter, Randomized, Open-label Phase 2 Study to Evaluate the Efficacy and Safety of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria（PNH）
This study is testing a new drug called HSK39297 to see if it can help adults with paroxysmal nocturnal hemoglobinuria who haven't tried other treatments feel better and improve their blood levels over 24 weeks.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 45 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Haisco Pharmaceutical Group Co., Ltd. Industry-sponsored |
| Locations | 1 site (Nanjing, Jiangsu) |
| Trial ID | NCT06561841 on ClinicalTrials.gov |
What this trial studies
This multicenter, randomized, open-label phase 2 study aims to evaluate the efficacy and safety of HSK39297 in adult patients diagnosed with paroxysmal nocturnal hemoglobinuria (PNH) who have not previously received complement inhibitor therapy. Participants will be treated with HSK39297 for a duration of 24 weeks. The study focuses on patients with elevated blood lactate dehydrogenase (LDH) levels and low hemoglobin levels, assessing the drug's impact on these parameters.
Who should consider this trial
Good fit: Ideal candidates for this study are adults aged 18 and older with a confirmed diagnosis of PNH who have not received prior complement inhibitor treatment.
Not a fit: Patients with hereditary or acquired complement deficiencies, active immunodeficiencies, or significant comorbidities may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a new therapeutic option for patients suffering from PNH, potentially improving their quality of life and health outcomes.
How similar studies have performed: While this approach is being evaluated in this specific context, similar studies have shown promise in treating PNH with novel therapies.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: 1. Male and female participants ≥ 18 years of age; 2. Diagnosis of PNH based on flow cytometry with clone size \> 10% by granulocytes; 3. Have not received complement inhibitor treatment; 4. Blood lactate dehydrogenase(LDH) values \> 1.5 ×upper limit of the normal range (ULN) ; 5. Hemoglobin level \< 100 g/L during the screening period. Exclusion Criteria: 1. Hereditary or acquired complement deficiency; 2. Active primary or secondary immunodeficiency; 3. History of splenectomy, bone marrow/ hematopoietic stem cell or solid organ transplants; 4. History of recurrent invasive infections caused by encapsulated organisms( e.g. meningococcus or pneumococcus) or Mycobacterium tuberculosis; 5. Patients with laboratory evidence of bone marrow failure (reticulocytes \< 100x10\^9/L, or platelets \< 30x10\^9/L or neutrophils \< 0.5x10\^9/L) ; 6. Active systemic infection within 2 weeks prior to study drug administration; 7. History of serious comorbidities that have been determined to be unsuitable for participation in the study. 8. Pregnant or Lactating women.
Where this trial is running
Nanjing, Jiangsu
- The First Affiliated Hospital of Nanjing Medical University — Nanjing, Jiangsu, China (Recruiting)
Study contacts
- Study coordinator: Fangqiong Li
- Email: lifangq@haisco.com
- Phone: +8602867258840
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.